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Sideroblastic anemia 3

MONDO:0014804

Also known as: GLRX5-related sideroblastic anaemia, GLRX5-related sideroblastic anemia, SIDBA3, adult-onset autosomal recessive sideroblastic anaemia, adult-onset autosomal recessive sideroblastic anemia, anemia, sideroblastic, 3, pyridoxine-refractory

22 clinical trials for this condition and its sub-types, 0 tagged with Sideroblastic anemia 3 itself.

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Part of

↑ Autosomal recessive sideroblastic anemia (2) ↑ Mitochondrial substrate carrier disorder (0)

Including sub-types (22) Tagged with Sideroblastic anemia 3 (0)

Trials to join now! 9 Not yet recruiting 3 Not yet finished but already full! 5 Completed 4 Terminated 1

New drug aims to tame hard-to-control seizures in rare mitochondrial disorders

Disease control Stopped early

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

Phase 2/3 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:03 UTC